Study in a Sentence: Researchers from the Institute for Bioengineering of Catalonia developed a three-dimensional model using patient cells to study myotonic dystrophy, opening new opportunities for finding personalized and effective treatments against this rare disease.
Healthy for Humans: Myotonic dystrophy, the most common form of muscular dystrophy, is a hereditary degenerative neuromuscular disease that affects about 1 in 8,000 people worldwide. There is currently no cure or specific treatment for myotonic dystrophy, and developing new treatments is difficult due to variation in clinical presentation and patient responses to intervention.
Redefining Research: This novel bioengineered 3D skeletal muscle model for myotonic dystrophy was fabricated using human cells and can be composed with cells from patients with different muscle diseases to create patient-specific models. To evaluate the functionality of this model, the researchers applied the microRNA drug AntagomiR-23b, which has previously shown to have effects on specific muscle cell functions implicated in myotonic dystrophy. They found that the molecular and structural characteristics of the muscle fibers derived from the patient recovered to their normal parameters when treated, demonstrating that this model may be useful in drug development for myotonic dystrophy therapies.